Natural History of the Progression of Choroideremia Study

NightstaRx Ltd, a Biogen Company319 enrolled

Overview

The objective of this natural history study is to gain a better understanding of the progression of choroideremia (CHM) and add to the knowledge base for this rare disease.

This study was previously posted by NightstaRx Ltd. In October 2020, sponsorship of the trial was transferred to Biogen.

Study Type

OBSERVATIONAL

Enrollment

319

Conditions

Choroideremia

Eligibility

Sex: MALEMin age: 18 Years

Medical Language ↔ Plain English

Key Inclusion Criteria: * Are willing and able to provide informed consent for participation in the study. * Have a clinical phenotype and confirmed genetic diagnosis of CHM. * Have active disease clinically visible within the macular region. * Are willing and able to undergo ophthalmic examinations once every 4 months for up to 20 months. * Have a BCVA better than or equal to 6/60 (20/200; decimal 0.1; LogMAR 1.0; 34-38 ETDRS letters) in at least one eye. Key Exclusion Criteria: * Have a history of amblyopia in the eligible eye. * Have any other significant ocular or non-ocular disease/disorder in the eligible eye which, in the opinion of the investigator, may put the subject at risk because of participation in the study, influence the results of the study or influence the subject's ability to participate in the study. * Have participated in an interventional research study in the past 6 months. NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Locations (20)

Research Site

Los Angeles, California, United States

Research Site

Miami, Florida, United States

Outcomes

Primary Outcomes

Change in Best-Corrected Visual Acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study (EDTRS)

BCVA will be assessed for both eyes using the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity (VA) chart. BCVA test should be performed prior to pupil dilation, and distance refraction should be carried out before BCVA is measured. Initially, letters are read at a distance of 4 meters from the chart. If \<20 letters are read at 4 meters, testing at 1 meter should be performed. BCVA is to be reported as number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters) in the study eye. The lower the number of letters read correctly on the eye chart, the worse the vision (or visual acuity). An increase in the number of letters read correctly means that vision has improved.

Time frame: Up to Month 20

Secondary Outcomes

Change from Baseline in Reading Performance using International Reading Speed Texts (IReST)

Reading performance will be evaluated prior to pupil dilation.

Time frame: Baseline up to Month 12

Change from Baseline in Color Vision

Colour vision will be tested prior to pupil dilation. Each eye will be tested separately and in the same order.

Time frame: Baseline up to Month 12

Change from Baseline in Visual Fields

The progression of defects in visual fields will be assessed in both eyes using perimetry equipment.

Time frame: Baseline up to Month 12

Change from Baseline in Contrast Sensitivity

Contrast sensitivity will be measured for both eyes, prior to pupil dilation, using a Pelli Robson chart.

Time frame: Baseline up to Month 12

Change from Baseline in Retinal Thinning Using Spectral Domain Optical Coherence Tomography (SD-OCT)

Data from ClinicalTrials.gov

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